PB2179: RETROSPECTIVE ANALYSIS OF THE EFFICACY AND SAFETY OF RUXOLITINIB IN CHRONIC GRAFT VERSUS HOST DISEASE (CGVHD). A SINGLE-INSTITUTION EXPERIENCE.
Authors: Ó. Muñiz, T. Torrado Chedas, A. Nieto Vazquez, A. Santamaria Lopez, E. Winsnes, L. González Rodríguez, C. Albo Lopez
Journal: HemaSphere (2022)

Background: GvHD still remains the main cause of transplant-related morbidity and mortality. While the first line treatment is corticosteroids, 50% of patients will not achieve and adequate response. At this moment, there is no consensus about which treatment should be used as second line and beyond. Because of this, the use of ruxolitinib has been positioning itself as a salvage therapy due to the latest results in different studies as well as the safety profile that offers compared with other alternative therapies. Aims: Evaluate the overall response rate (ORR) at 6 and 12 months and the adverse eventes (AEs) of ruxolitinib used as salvage therapy for cGvHD. Methods: In this single-center retrospective analysis we reviewed the charts of patientes who received ruxolitinib for cGvHD between January 2017 and December 2021 in Hospital Álvaro Cunqueiro, Vigo. Statistical analysis have been made by using SPSS Statistics. Staging of cGvHD was based on National Institutes of Health (NIH) disease severity score. AEs were grading according to Common Terminology Criteria (CTCAE) version 5.0. Results: Our cohort comprised 14 patients. Median patient age was 54 years and 78.6% (n=11) were male. Most common diagnosis for undergo hematopoietic stem cell transplantation (HSCT) was myelodysplastic syndrome (35.7%, n=5). Conditioning regimen was reduced intensity in 78.6% (n=11) and myeloablative in 24.6% (n=3). The average onset of cGvHD was 21 months post-HSCT, and the mediant time to ruxolitinib initiation was 44 months. Almost all patients had moderate (71.4%, n=10) or severe (21.4%, n=3) disease. Most common involved organ was skin in 64.3% (n=9). On average, patients received 3 previous therapies before ruxolitinib. Among previous treatments, predonisone (92.9%, n=13), tacrolimus (50%, n=7), sirolimus (50%, n=7) and photoapheresis (35.7%, n=5) were the most frequently used. 57.1% (n=8) of patients were able to reduce (n=2) or stop (n=4), and stop other inmunosupresor (n=2). After 6 months, we observed an ORR of 64.3% (n=9) with complete response (CR) in 35.7% and partial response (PR) in 28.6%. After 12 months, ORR was observed in 42.8% (n=6), with CR in 35.7% and PR in 7.1%. At 1 year, treatment failure was observed in 35.7% (n=5). Causes of failure were progression of cGvHD (n=4) and death in 1 of them. The most common AEs were cytopenias, affecting 71.4% of patientes (n=10), but only 21.4% were grade 3-4. The most frequent cytopenia was anemia in 42.8% (n=6), followed by neutropenia (35.7%, n=5) and thrombocytopenia (14.2%, n=2). 42.8% (n=6) developed a total of 9 infectious complications requiring admissión, most commonly pneumonia. The infectious complications caused death in 14.3% (n=2). Other AEs were: urinary infections not requiring admissión (n=2), cytomegalovirus viremia (n=1), herpes simplex virus reactivation (n=1), deep venous thromboembolism (n=1), diarrhea (n=1), skin atrophy (n=2) and folliculitis (n=2). None of those AEs caused ruxolitinib discontinuation. Summary/Conclusion: In our analysis, the ORR obtained have been greater than 50%, maintaining this response at one year of therapy. Besides its effectiveness, ruxolitinib favors dose reducing or discontinuation in corticodependent patientes. Ruxolitinib is a well-tolerated drug with a good safety profile. Globally, 2 patientes died of complications (14.3%), which is a low mortality rate in fragile and heavily pre-treated patients.